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International Alliance of ALS/MND Associations

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Expanded Access

Every medication or medical device used today underwent extensive testing to ensure its safety and efficacy. The United States set the standard for clinical trials more than 60 years ago, and the process has not changed much since, except to become more complex and lengthy. It can take a decade or more for a drug candidate to reach the clinic, which for many people is too long to wait.

Fortunately, over the past decade, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have taken steps to expedite certain aspects of clinical trials to more quickly deliver important drugs to people in need. In the US, the expanded access pathway (also called “passionate use”) gives people with life-threatening illness early access to drugs that have not finished clinical testing and, in turn, have not yet been approved by the FDA. The EMA supports expanded access and the programs are operated independently by each member of the European Union.

The Conditions for Compassionate Use

Since compassionate use programs involve giving people drugs that have are not proven safe and effective, the FDA always prefers that people participate in a clinical trial first. The FDA and other regulatory agencies take careful steps to make sure they are not putting patients under undue risk. The FDA only qualifies drugs for expanded access available if:

  • The people who would take it have a immediately life-threatening illness and there are no comparable or effective treatments already available to them
  • These people cannot enroll in a clinical trial, whether it is because no trial is available or the are not eligible for any trials
  • The potential benefits of taking the drug outweigh the potential risks
  • Giving the drug to people will not interfere with an ongoing clinical trial

Expanded access comes in many forms. In some cases, the program is designed to bridge the gap between the end of a clinical trial and marketing approval for a large group of people. In other cases, the pathway is used for a smaller group of people who need a drug that is not actively being developed for clinical use. Finally, a doctor may get permission to give an individual an investigational drug in an emergency setting.

Expanded Access for People with ALS/MND

ALS/MND’s status as a severe, life-threatening disease with no effective treatments might qualify certain experimental ALS/MND drugs for expanded access programs.

 

The Risks Associated with Expanded Access Programs

While the FDA and EMA fully support expanded access programs, they prefer that people enter clinical trials because clinical research offer people more protection if the treatment is unsafe or ineffective. Also, clinical trials are the best way to prove a medication’s safety and efficacy.

It’s important to remember that drugs made available as part of an expanded access program have not been approved for use in the clinic — researchers have not yet proven that it is safe and effective. These drugs make produce unexpected side effects. It is important to discuss the risks with your doctor.

Learn more about expanded access from the FDA here.

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Advocacy

  • Advocacy Toolkit

  • Marco Antonio Alvarez Mercado, Mexico

    Marco Antonio Alvarez Mercado, Mexico

  • Dad

    Dad

  • Philip Brindle,  MND Association,  Diagnosed 2015,  England

    Philip Brindle, MND Association, Diagnosed 2015, England

  • Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

    Soledad Rodriguez, FUNDELA, Diagnosed 2013, Spain

  • Christian Bär, Germany

    Christian Bär, Germany

  • Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

    Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

  • Sam Hayden-Harler, Motor Neurone Disease (MND) Association, UK

    Sam Hayden-Harler, Motor Neurone Disease (MND) Association, UK

  • Rosie Riley, Les Turner ALS Foundation, USA

    Rosie Riley, Les Turner ALS Foundation, USA

  • Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

    Laurie Petit-Jean, Diagnosed 2012 , ARSLA, France

  • Denis Blais, Diagnosed 2015 , ALS Canada

    Denis Blais, Diagnosed 2015 , ALS Canada

  • Margreth Burger-Saile, Diagnosed 2011,  ALS Schweiz,  Switzerland

    Margreth Burger-Saile, Diagnosed 2011, ALS Schweiz, Switzerland

  • Timmy, ALS Liga

    Timmy, ALS Liga

  • Frank "Papa" Taylor, USA

    Frank “Papa” Taylor, USA

  • David Bishop

    David Bishop

  • Fernando Ocampo Cardona, Colombia

    Fernando Ocampo Cardona, Colombia

  • Ian and Teresa Roberts

    Ian and Teresa Roberts

  • H. Todd Kelly, Diagnosed 2013 , ALS Hope Foundation, USA

    H. Todd Kelly, Diagnosed 2013 , ALS Hope Foundation, USA

  • Josée Kolijn-de Man, Diagnosed 2015 , ALS Patients Connected, The Netherlands

    Josée Kolijn-de Man, Diagnosed 2015 , ALS Patients Connected, The Netherlands

  • England-Lee-Millard, UK

    England-Lee-Millard, UK

  • Marcel R. Wernard, Diagnosed 2016,  ALS Patients Connected,  The Netherlands

    Marcel R. Wernard, Diagnosed 2016, ALS Patients Connected, The Netherlands

  • 727747090571358167

    727747090571358167

  • Alberto Baez Murillo, Colombia

    Alberto Baez Murillo, Colombia

  • Tammy Moore and Eddy Lefrancois

    Tammy Moore and Eddy Lefrancois

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Alejandro Aquino, Diagnosed 2011 , Asociación ELA Argentina

    Alejandro Aquino, Diagnosed 2011 , Asociación ELA Argentina

  • Valdomiro Xavier Honório, Brazil

    Valdomiro Xavier Honório, Brazil

  • Claudia Cominetti, Associazione conSLAncio Onlus,  Italy

    Claudia Cominetti, Associazione conSLAncio Onlus, Italy

  • Karl Hughes, Diagnosed 2010 , IMNDA,  Ireland

    Karl Hughes, Diagnosed 2010 , IMNDA, Ireland

  • Erwin Coppejans, Diagnosed 2007 , ALS Liga België, Belgium

    Erwin Coppejans, Diagnosed 2007 , ALS Liga België, Belgium

  • Aida Trzmiel de Guterman, Asociacion ELA Argentina, Diagnosed 2007, Argentina

    Aida Trzmiel de Guterman, Asociacion ELA Argentina, Diagnosed 2007, Argentina

  • Jon Newsome, USA

    Jon Newsome, USA

  • Mahmood Anwar, UK

    Mahmood Anwar, UK

  • Leon Ryba, Argentina

    Leon Ryba, Argentina

  • Liam Dwyer, England

    Liam Dwyer, England

  • Michael Lee, Australia

    Michael Lee, Australia

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • Guoqiang Xu, Diagnosed 2016 , Shaanxi ALS Association, China

    Guoqiang Xu, Diagnosed 2016 , Shaanxi ALS Association, China

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
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  • Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • Eddy LeFrançois, Diagnosed 1992,  ALS Canada

    Eddy LeFrançois, Diagnosed 1992, ALS Canada

  • 393647_2252248542053_984912751_n

    393647_2252248542053_984912751_n

  • Yessenia Hernandez Mendoza, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Yessenia Hernandez Mendoza, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • IMG_2658

    IMG_2658

  • Fabrice Kamp, Germany

    Fabrice Kamp, Germany

  • Irene McCaughey, Diagnosed 2011,  MND Australia

    Irene McCaughey, Diagnosed 2011, MND Australia

  • Osiel Mendoza, Diagnosed 2016 ,  ALS Therapy Development Institute, USA

    Osiel Mendoza, Diagnosed 2016 , ALS Therapy Development Institute, USA

  • Chun Ju Xiao, China

    Chun Ju Xiao, China

  • Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

    Sharon Corosanite, Diagnosed 2014 , ALS Hope Foundation, USA

  • Art Eggert, USA

    Art Eggert, USA

  • Richard Clark, MND New Zealand,  Diagnosed 2011

    Richard Clark, MND New Zealand, Diagnosed 2011

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